Finally, First Cancer Gene Therapy in the U.S. Approved by FDA

For the first time in the medical history of the United States, the Food and Drug Administration (FDA) issued its approval of a cancer gene therapy that could potentially treat children and young adults who have leukemia.

According to CNN, the treatment aims to give patients a second chance in worst cases when the first line of drugs or cancer treatment failed. FDA referred to it as the first cancer gene therapy to be available in the country.

Further reports claimed that the therapy is designed “to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults.”

The CAR-T (chimeric antigen receptor T cell) cancer gene therapy will be marketed as Kymriah (tisagenlecleucel) by Novartis International AG, a multinational pharmaceutical company based in Basel, Switzerland. Novartis is considered as one of the leading pharmaceutical companies when it comes to innovative medicine.

Referring to the FDA approval, Joseph Jimenez, CEO of Novartis said in a statement published on the company’s website:

“At Novartis, we have a long history of being at the forefront of transformative cancer treatment. Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need. With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care.”

The Cancer Gene Therapy ‘Kymriah’

What exactly is Kymriah and how does this revolutionary cancer gene therapy work?

Kymriah is a customized treatment that uses a cancer patient’s own T cells. The T cells are extracted from the patient and will be cryogenically frozen. The frozen cells will then be transported to a manufacturing facility at Novartis Morris Plains, New Jersey.

At the manufacturing facility, the T cells will be genetically altered to create a new gene that codes for protein, called chimeric antigen receptor (CAR). The new gene will direct the T cells to target and kill leukemia cells with a specific antigen on their surface. Afterwards, the genetically modified cell will be infused back into the patient.

FDA’s Approval and Results of the ELIANA Trial

FDA’s approval of Kymriah is based on the results of the pivotal open-label, multicenter, single-arm Phase II ELIANA trial.

The ELIANA trial is considered the first pediatric global CAR-T cell therapy registration trial which involves 68 patients suffering from a type of acute lymphoblastic leukemia in 25 centers across the United States, Europe, Canada, Australia, and Japan.

63 out of the 68 patients that went through the cancer gene therapy trial were found evaluable for efficacy. Results have shown that 52 out of the 63 patients or an astounding 83 percent of the subjects who received Kymriah treatment achieved complete remission (CR) or CR with incomplete blood count recovery (CRi) within three months of infusion.

At six months, 89 percent of the patients who received the cancer gene therapy were still alive, and at 12 months, 79 percent had survived the trial.

Aside from that, researchers and doctors involved in the trial didn’t find any sign of minimal residual disease (MRD), which is a blood marker that indicates potential cancer relapse, among the patients.

Carl June, MD, the Richard W. Vague Professor of Immunotherapy and Director of the Center for Cellular Immunotherapies in University of Pennsylvania’s Perelman School of Medicine, who is also a pioneer of this new treatment said:

“This therapy is a significant step forward in individualized cancer treatment that may have a tremendous impact on patients’ lives. Through our collaboration with Novartis, we are creating the next wave of immunocellular cancer treatments, and are eager to progress CAR-T therapy in a host of hematologic and other cancer types.”

The Cost of a One Time Cancer Gene Therapy

Novartis will sell the one-time cancer gene therapy for a staggering $475,000 USD. An amount which Patients for Affordable Drugs‘ founder David Mitchell cited as ‘excessive.’

In a statement released through the Patients for Affordable Drugs’ website, Mitchell claimed that American taxpayers invested over $200 million USD in CAR-T’s discovery. A part of the statement read:

“But let’s remember, American taxpayers invested over $200 million in CAR-T’s discovery. To date, Novartis has not acknowledged the significance of taxpayers’ investment, and the company declined to detail its own investment.

While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive. Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more.

The drug pricing system in America is completely broken. Until policy in this country changes, the vicious cycle of patients struggling under high drug prices will continue.”

Just recently, the group had a meeting with Novartis to negotiate the price of the treatment. The results of the meeting have not been made public yet. To date, other pharmaceutical companies such as Kite Pharma and Juno Therapeutics are developing their own cancer gene therapy.

In fact, Kite Pharma is now awaiting approval from FDA for its CAR-T therapy to treat a form of blood cancer in adults.

On average, it takes about 22 days to create a Kymriah therapy, from the removal of the T cells from the patient to when they are infused back into the patient’s body. The question now is how Novartis will be able to manufacture personalized therapies quickly enough to get them to patients across the country.

Within a month, Kymriah will be made available to 20 US hospitals, but Novartis said that 32 sites in total would offer the treatment in the coming months.

Do you agree that $475,000 USD is too much for a one-time cancer gene therapy? Let us know your thoughts in the comment section below.